A Bayesian adaptive design for biomarker trials with linked treatments

British Journal of Cancer

Volumn 113, Issue 5, 2015, Pages 699-705

  Wason, James M S ^a   Abraham, Jean E ^b,c,d   Baird, Richard D ^b,c,d   Gournaris, Ioannis ^b   Vallier, Anne Laure ^c   Brenton, James D ^b,d   Earl, Helena M ^b,c,d   Mander, Adrian P ^a  

^a MRC BIOSTATISTICS UNIT   (United Kingdom)

^b UNIVERSITY OF CAMBRIDGE   (United Kingdom)

^c CAMBRIDGE UNIVERSITY HOSPITALS NHS FOUNDATION TRUST   (United Kingdom)

^d Cambridge Experimental Cancer Medicine Centre   (United Kingdom)

Author keywords

adaptive design; adaptive randomisation; Bayesian; biomarkers; phase II; stratified medicine

Indexed keywords

BIOLOGICAL MARKER; ANTINEOPLASTIC AGENT; TUMOR MARKER;

ARTICLE; BAYES THEOREM; BAYESIAN ADAPTIVE RANDOMIZATION; CONTROL GROUP; CONTROLLED STUDY; EXPERIMENTAL THERAPY; HUMAN; MOLECULARLY TARGETED THERAPY; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; PROBABILITY; RANDOMIZATION; RANDOMIZED CONTROLLED TRIAL; STUDY DESIGN; TREATMENT RESPONSE; METABOLISM; NEOPLASMS; PHASE 2 CLINICAL TRIAL (TOPIC); PROCEDURES; RANDOMIZED CONTROLLED TRIAL (TOPIC); STATISTICAL ANALYSIS; TREATMENT OUTCOME;

ANTINEOPLASTIC AGENTS; BAYES THEOREM; BIOMARKERS, TUMOR; CLINICAL TRIALS, PHASE II AS TOPIC; DATA INTERPRETATION, STATISTICAL; HUMANS; NEOPLASMS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; TREATMENT OUTCOME;

EID: 84940792112     PISSN: 00070920     EISSN: 15321827     Source Type: Journal    
DOI: 10.1038/bjc.2015.278     Document Type: Article

References (22)

1. Adams CP, Brantner VV (2006) Estimating the cost of new drug development: is it really $802 million? Health Aff 25(2): 420-428.
2. Barker AD, Sigman CC, Kelloff GJ, Hylton NM, Berry DA, Esserman LJ (2009) I-SPY 2: an adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clin Pharmacol Ther 86(1): 97-100.
3. Berry SM, Carlin BP, Lee JJ, Muller P (2010) Bayesian adaptive methods for clinical trials. CRC press.
4. Cai J, Xu L, Tang H, Yang Q, Yi X, Fang Y, Zhu Y, Wang Z (2014) The role of the PTEN/PI3K/Akt pathway on prognosis in epithelial ovarian cancer: a meta-analysis. Oncologist 19(5): 528-535.
5. Chapman PB, Hauschild A, Robert C, Haanen JB, Ascierto P, Larkin J, Dummer R, Garbe C, Testori A, Maio M (2011) Improved survival with vemurafenib in melanoma with BRAF V600E mutation. N Engl J Med 364(26): 2507-2516.
6. Etemadmoghadam D, Beroukhim R, Mermel C, George J, Getz G, Tothill R, Okamoto A, Raeder MB, Harnett P, Lade S (2009) Integrated genomewide DNA copy number and expression analysis identifies distinct mechanisms of primary chemoresistance in ovarian carcinomas. Clin Cancer Res 15(4): 1417-1427.
7. Etemadmoghadam D, Weir BA, Au-Yeung G, Alsop K, Mitchell G, George J, Davis S, DGÇÖAndrea AD, Simpson K, Hahn WC (2013) Synthetic lethality between CCNE1 amplification and loss of BRCA1. Proc Natl Acad Sci 110(48): 19489-19494.
8. Freidlin B, Korn EL (2013) Adaptive randomization versus interim monitoring. J Clin Oncol 31(7): 969-970.
9. Gallo P (2006) Operational challenges in adaptive design implementation. Pharm Stat 5(2): 119-124.
10. Jimeno A, Messersmith WA, Hirsch FR, Franklin WA, Eckhardt SG (2009) KRAS mutations and sensitivity to epidermal growth factor receptor inhibitors in colorectal cancer: practical application of patient selection. J Clin Oncol 27(7): 1130-1136.
11. Kaplan R, Maughan T, Crook A, Fisher D, Wilson R, Brown L, Parmar M (2013) Evaluating many treatments and biomarkers in oncology: a new design. J Clin Oncol 31(36): 4562-4568.
12. Kim ES, Herbst RS, Wistuba II, Lee JJ, Blumenschein GR, Tsao A, Stewart DJ, Hicks ME, Erasmus J, Gupta S (2011) The BATTLE trial: personalizing therapy for lung cancer. Cancer Discov 1(1): 44-53.
13. Kwak EL, Bang YJ, Camidge DR, Shaw AT, Solomon B, Maki RG, Ou SH, Dezube BJ, Jänne PA, Costa DB (2010) Anaplastic lymphoma kinase inhibition in non-small-cell lung cancer. N Engl J Med 363(18): 1693-1703.
14. Lee JJ, Chen N, Yin G (2012) Worth adapting? Revisiting the usefulness of outcome-adaptive randomization. Clin Cancer Res 18(17): 4498-4507.
15. Lee JJ, Gu X, Liu S (2010) Bayesian adaptive randomization designs for targeted agent development. Clin Trials 7(5): 584-596.
16. Papadimitrakopoulou V, Wistuba II, Lee JJ, Tsao AS, Kalhor N, Fossella FV, Heymach J, Alden CM, Gettinger SN, Kevin R (2013) BATTLE-2 program: a biomarker-integrated targeted therapy study in previously treated patients with advanced non-small cell lung cancer (NSCLC). J Clin Oncol 31(suppl): abstr TPS8118.
17. Peeters M, Douillard JY, Van Cutsem E, Siena S, Zhang K, Williams R, Wiezorek J (2013) Mutant KRAS codon 12 and 13 alleles in patients with metastatic colorectal cancer: assessment as prognostic and predictive biomarkers of response to panitumumab. J Clin Oncol 31(6): 759-765.
18. Stewart DJ, Kurzrock R (2009) Cancer: the road to Amiens. J Clin Oncol 27(3): 328-333.
19. Wason J, Trippa L (2014) A comparison of Bayesian adaptive randomization and multi-stage designs for multi-arm clinical trials. Statist Med 33(13): 2206-2221.
20. Wason JM, Stecher L, Mander AP (2014) Correcting for multiple-testing in multi-arm trials: is it necessary and is it done? Trials 15(1): 364.
21. Wason JMS, Jaki T, Stallard N (2013) Planning multi-arm screening studies within the context of a drug development program. Statist Med 32(20): 3424-3435.
22. Yap TA, Sandhu SK, Workman P, de Bono JS (2010) Envisioning the future of early anticancer drug development. Nat Rev Cancer 10(7): 514-523.